CBER Data Standards Management

Presented to:DC CDISC Group

April 25, 2014

Agenda• CBER Data Standards Management• CBER Data Standards Project Portfolio• Focus for Addressing Mandates and Processing Gaps

for CDISC Data Standards

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Role of Data Standards• FDA recognizes the need for standards to support standardization of

industry regulatory submissions and to enable data reuse and cross reference throughout a product’s life cycle

• Data standards support the need for functional interoperability across FDA core functions which is simplified as:

“Data contained in disparate regulatory submissions is reused to support another regulatory function without having to understand how the source data was collected or the need to log into different systems to validate the same information”

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Data Standards Groups

• Representatives are Center/Agency SMEs: Electronic Health Record and Health Information Technology (HIT) Standards

• Representatives are Center/Agency SMEs: CDISC, HL7, ICH, ISO

• Center SME liaisons to FDA Data Standards Council and FDA Standards Management Group

• Center Subject Matter Experts (SMEs)that are repurposed for multiple internal/external standards activities Center Data

Standards Groups: DSS, CDER DSPB

Agency Data Standards

Groups

HHS OPDIV Collaboration

s and ONC workgroups

International and /External

Standards Development Organizations

(SDOs)

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CBER Data Standards Subcommittee (DSS)

• Provides a formal governance and project management process for data standards projects with interdependencies with FDASIA and user fee program goals for:

– IT systems, especially support for structured electronic regulatory submissions

– Business process changes as a result of new tools and systems– Regulatory policy changes: new or updated industry guidance to

use the data standards– Data standards: development, testing, adoption and

implementation

• Projects are categorized by a Data Standards Lifecycle Phase

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CBER DSS Governance Relationships• CBER Information Management Coordinating Committee (IMCC) oversight ensures

that DSS projects align with CBER’s IT Strategic Plan goals for projects that have IT interdependencies:

1. Information Management

2. Innovation

3. Partnerships

• CBER Review Management Coordinating Committee (RMCC) coordination ensures that DSS projects align for interdependencies with CBER business processes and regulatory policies

• Center-level informatics coordination ensures that DSS projects align with Agency/Center priorities and goals pertaining to Regulatory Science and Research

CBER Data Standards Portfolio• Active projects are near term activities that require CBER review

experts and IT resources that must be accounted for (e.g., project plans and budget submissions for IT systems or contractor support)

• Some projects pertain to other Agency/non-CBER initiatives that require close monitoring, e.g., FDASIA or MDUFA

• Projects are grouped by data standards lifecycle phase and include touch points for:– IT systems– Regulatory policy/guidance documents– Business process changes– Interactions with other internal/external groups 7

CBER Standards Portfolio by Working Group, Lifecycle Phase and IT Strategic Goal Area

Data Standards SubcommitteeCBER CDIS

C

Development & Testing

CDISC Data Submission

XML Pilot

Post Market Submissions

Implementation

eVAERS / ICSR

(IT, Regulatory Policy,

Business Process)

Adoption

ICH E2B(R3) IDMP

Adoption (for ICSR)

FDA IDMP

Adoption

ISO IDMP Implementatio

n Guides (ISO 11238)

ISO IDMP Maintenance

FDA SRS Redesign

Product Dictionary/

Integrity

Regulated Product

Submissions

Development & Testing

IMDRF: RPS for Medical

DevicesICH M8: eCTD v4.0 Project

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Operations and Maintenance Phase

Operations and Maintenance Activity

Electronic Submissions Formats

SDTM 3.1.1

through 3.1.3

ICH E2B-R2

SPL ADaM 2.1 eCTD

Regulatory Policy & Guidance Documents

eLDD eCTD

eSubs & FDASIA

eStudy Data

Study Data

Standards Technical

Guide

eSRR

Reviewer/Industry Training and Support

JReview Dataset

s loading request

s

TrainingCBER

Website, SOPs

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Performance monitoring helps to identify problems and issues from the review community – Process improvements help to address regulatory requirements for data reuse and linking, e.g., pre and post market safety reports

Center Informatics Monitoring Projects

Data Standards Monitoring ActivityExternal SDO Work

CDISC

CDASH

TA Standards Initiative Project

Plan

SDTM 3.1.4

HL7

RCRIM RPS, SPL

Pharmacy:

Common Product Model

Public Health

Reporting

Electronic Health

Records

ISO TC 215 Workgroup 6

ICSR Technical

ReportIDMP

Standards

IMDRF

IMDRF Device Table of

Contents

Resources are repurposed for multiple projects but still cannot monitor everything we know about. Some review SMEs also monitor external groups: e.g., PhUSE, CPATH, CFAST, TransCelerate BioPharma

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CBER Data Standards Challenges

• CBER is relatively small Center and we have a very diverse product portfolio:– NDA– BLA– 510(K)– PMA

• CBER must account for a wider variety of data standards applicability to different regulatory/legislative drivers which may not overlap– FDA– CDC

• Requires CBER to work more collaboratively internally and externally– Regulatory Use Cases– Public Health Research and Reporting 11

Mandates and Submissions Processing Gaps

• PDUFA commitments state that FDA will require electronic submission of study data by 2017; however

• CBER has identified several support gaps that must be addressed before this can be accomplished:

– Understanding the business impact based upon review workload– Addressing known errors and inconsistencies in standards adoption– Understanding training requirements by review discipline– Closing the gap between development and implementation based upon

tangible FDA business needs– Automation of submission receipt tracking and validation processes

• Subject to other User Fee Programs: BsUFA, MDUFMA, GDUFA12

PDUFA Business Impact by Review Workload

IND NDA BLA TOTAL 2131 37 766 2934

PDUFA WORKLOAD2004 4 169 2177

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Number of CDISC Submissions by CY

JAN-DEC JAN-APR2012 2013 2014 TOTAL

Unknown 30 20 50*Based upon file validation tracking

Submissions by Product TypeType ADaM SDTM TOTAL

Vaccines 1 2 3Blood

Derivatives17 30 47

Cell/Tissue/Gene Therapy

0 0 0

*Allergenic products are exempt

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Standards Adoption and Use: Top 7 Errors

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Error Type Description CDISC Validator Business Rules

Consistency Inconsistent value for standard units of measurement

SD0007

Presence NULL value in variable marked as required

SD002

Terminology Value for AECONTRT not found in (NY) CT codelist

CT0070

Metadata Dataset variable not defined in define.xml

SD0059

Coss-reference Invalid ARM/ARMCD SD0071

Format Invalid ISO 8601 value SD1011

Limit Negative value for --DUR SD0015

Near Term Activities• Consistent project management process for CBER CDISC

Workgroup:

– Focus on managing activities by data standards lifecycle to meet agency mandates

– Focus on meeting specific review needs by review discipline• Slow down development and monitoring tasks

– Address long term training needs– Implement formal process for adoption and migration planning

• Limit portfolio projects to what we can manage with operational excellence

– Focus on addressing the operational issues with standards use by reducing errors and providing clear guidance (internal and external) about the data needed for review and data reuse

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Background Slides

DC-CDISC Group MeetingApril 25, 2014

Data Standards Lifecycle PhasesDevelopment: Create new or major revision of existing standard to address a specific regulatory need: data exchange, content, terminology. Includes SDO balloting and/or collaboration with internal/external stakeholders

Testing: Simulate regulatory use case by applying the standard to the business problem. Includes creation of test tools used by industry and/or FDA reviewers. Assessment of performance and success criteria

Adoption: Center “go/no go” decision based upon testing results and success criteria

Implementation: Execute processes to integrate standard into business practice: review, IT, regulatory policy

Operations and Maintenance: Leverage CBER CCB process to maintain systems for continued standards use

Performance Monitoring: Ensure standard continues to meet business needs and adjust to changing environment

Engage SDO: If standard no longer meets CBER requirements, propose major revision or new standards project

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CBER DSS Standards PortfolioData standards categories are used to help determine resource requirements for each project:

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Standards Type Definition ExampleData Exchange Content and format in

which particular types of data are to be presented and exchanged. Data exchange standards include file format specifications

Structured Product Labeling (SPL)

Data Format Structure, content, naming conventions, and variable formats for any given data domain

SAS transport files (SAS XPT)

Terminology Controlled vocabularies to improve communications and enhance analytical capabilities

Standardized terms for anatomical body sites

Data Standards Monitoring • DSS does not manage data standards monitoring activities• Monitoring activity still requires CBER resource commitments:

– CBER Subject Matter Experts in review offices and ADRM– Participation in meetings and teleconferences/webinars– Review of documents against FDA business requirements

• If an Agency, non-CBER or SDO project appears to be on an expedited timeframe that will have a future impact on CBER’s business, then projects are reprioritized and may be moved to the DSS active project portfolio:– CDRH: Unique Device Identifiers– CDER: CDISC Therapeutic Area Standards (TA)– ISO IDMP Implementation Guides

• Coordination is managed and tracked under the Center Informatics Group with updates to DSS, IMCC and RMCC as appropriate 20